One Step Closer To A “Smart” Stem Cell Therapy

August 16th, 2016 by FFB Canada

Today, there are no approved stem-cell treatments for blinding eye diseases. Dr. Andras Nagy’s research is trying to solve this problem by developing a safe and “smart” stem-cell therapy. To do this, his research team is combining gene therapy and stem cell therapy to generate specialized stem cells that have been genetically modified to release a sight-saving drug. As a result, these specialized, genetically-modified stem cells have the ability to replace dying cells, and also prevent further cell death.

Dr. Nagy is famous for making multiple major breakthroughs in regenerative medicine. First, he developed a technique to create stem cells from other cells of the body. Next, he developed a technique that manipulates how cells express different molecules. Last year, Dr. Nagy was awarded funding from the Foundation Fighting Blindness for his project that combines these techniques to develop a new “smart” stem cell therapy for age-related macular degeneration (AMD), the leading cause of blindness in people over the age of 50.

The vision loss that occurs during wet-AMD is caused by the growth of leaky blood vessels, which is caused by the over production of a protein called vascular endothelial growth factor (VEGF). Current treatments administer anti-VEGF drugs directly into the eyes, a process that requires regular (monthly or bimonthly) injections. Dr. Nagy’s team recently developed a new anti-VEGF drug which they call “VEGF Sticky-trap.” The team’s long-term objective is to use VEGF Sticky-trap to create a new “smart” cell-based therapy.

Although there a number of very effective anti-VEGF drug therapies to treat AMD, these treatments require regular injections into the eye, which can be a burden to patients and their families, particularly those who need to travel long distances. To help solve this problem, Dr. Nagy is working on a single “smart” cell therapy that would cure AMD with a single treatment of genetically-modified cells.

After one year of funding from the FFB, Dr. Nagy and his team are more optimistic than ever about their research, stating: “So far we have been successful in generating cells that can produce VEGF Sticky-trap. This is by far the safest approach to treat macular degeneration and other related diseases.”

We are very excited about Dr. Nagy’s progress, so we asked him: How much longer will it be before there are approved stem cell treatments for blinding diseases? And, what can we do to speed things up?

Dr. Nagy’s thoughtful answer points to the importance of working together to raise awareness about stem cell science and warns of the dangers of untested approaches. He explains: “It is only a matter of time until the first cell therapy clinical trials for eye diseases start in Canada. As clinical trials for cell therapy are very expensive, funding is absolutely crucial in accelerating progress. In addition, for researchers, thinking ahead early enough and getting the right people involved is an asset. For the public, it is important to raise awareness and understanding about stem cell science. Stem cell tourism and the use of unproven, unregulated and potentially dangerous therapies can add a burden to Canada’s health care system and, as a result, decrease funds available for regulated cell therapy.” Thank you to all of the FFB donors who are helping to drive this research forward.

We can’t wait to ask Dr. Nagy more questions about his research at the Toronto Vision Quest event on September 8, 2016. Register today.

You can also support Dr. Nagy’s and other incredible Canadian vision research right now:

Hi Bill,
We’re pretty excited about Dr. Nagy’s work too! Currently, his research is still focused in the laboratory and it is also focused on the disease age-related macular degeneration. There is excellent RP research happening on the west coast, such as the work by Dr. Orson Mortiz. Amazingly, one of Dr. Moritz’s graduate students also lives with RP. You can read about his project and meet Ruanne here: http://ffb.ca/can-valproic-acid-vpa-be-used-to-treat-retinitis-pigmentosa-the-answer-is-in-the-gene/

Julie

What about Stargardt disease? Will ee have some research for this too?

Hi Julie,
The Foundation Fighting Blindness also funds research that is more directly relevant to Stargardt disease. I recommend that you check out the excellent work by Dr. Gilbert Bernier, who is developing a method to restore sight by transplanting stem cells. You can read an overview of his research here: http://ffb.ca/restoring-vision-with-stem-cells/

Marc Hebert

Hi,
Great to hear that the research is moving forward with the stem cells & genetics in conjunction with the electrical/electronic marginal fixes. I would also be VERY INTERESTED in any opportunity for clinical research trials/programs in Canada or USA for RP patients. Any response would be greatly appreciated on this subject.
Marc Hebert – P. Eng.
Mechanical Engineer
RP patient – East Coast Canada
Moncton NB

Hi Marc,
Thanks for reaching out and sharing your interest about clinical trials. We will be sure to post any updates about new opportunities to become involved. I encourage you to learn more about the Foundation Fighting Blindness Patient Registry, which is a is a clinical database that connects people living with retinal eye diseases to emerging clinical trials and research: http://ffb.ca/research/patient-registry/

Marc Hebert

Thank you for the reply and registry info. I will register with your database and keep checking your updates & posts.

Marc Hebert

Thank you for the reply and registry info. I will register with your database and keep checking your updates & posts.